Wilson's disease gene therapy

Abstract

There is described a new gene therapy approach for treating Wilson's disease in which a nucleic acid molecule is used which comprises a nucleotide sequence encoding for a functional ATP7B protein wherein the nucleotide sequence has at least 85% identity to the sequence of SEQ ID NO: 1. Also described are vectors comprising the nucleotide sequence and methods and uses thereof.

The invention claimed is: 
     
       1. A nucleic acid molecule comprising a nucleotide sequence encoding for a functional ATP7B protein wherein the nucleotide sequence has the sequence of SEQ ID NO.1. 
     
     
       2. A vector for expressing ATP7B protein, the vector comprising the nucleic acid molecule of  claim 1 . 
     
     
       3. The vector of  claim 2 , further comprising a liver specific promoter. 
     
     
       4. The vector of  claim 2 , wherein the vector is an AAV vector. 
     
     
       5. The vector of  claim 2 , wherein the vector is a single stranded vector. 
     
     
       6. A host cell comprising the nucleic acid molecule of  claim 1 . 
     
     
       7. A pharmaceutical composition comprising the nucleic acid molecule of  claim 1 , and one or more pharmaceutically acceptable excipients. 
     
     
       8. A pharmaceutical composition comprising the vector of  claim 2 , and one or more pharmaceutically acceptable excipients. 
     
     
       9. A method of treating Wilson's disease comprising administering a therapeutically effective amount of the vector of  claim 2  to a patient suffering from Wilson's disease. 
     
     
       10. A method for delivery of a nucleotide sequence encoding ATP7B protein to a subject, which method comprises administering to the said subject the vector of  claim 2 .